Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Severe growth failure with primary insulin-like growth factor-1 deficiency

Initial treatment

The condition must be caused by severe primary insulin-like growth factor-1 deficiency (IGFD), with IGFD deficiency for the purpose of PBS subsidy defined as a basal IGF-1 level (measured any time prior to initiating treatment with this drug) below the 2.5th percentile adjusted for each of: (i) age, (ii) gender; AND

The condition must have resulted in the patient experiencing short stature, with short stature for the purpose of PBS subsidy defined as the patient's height (measured any time prior to initiating treatment with this drug) being at least 3 standard deviations below the norm, adjusted for each of: (i) age, (ii) gender; AND

Patient must have a growth velocity below the 25th percentile for bone age and sex measured over a 12 month interval (or a 6 month interval for an older child); AND

The condition must not be caused by growth hormone deficiency; AND

Patient must have a bone age of less than 13.5 years (females); or

Patient must have a bone age of less than 15.5 years (males); AND

The condition must not be caused by secondary causes of IGFD - prior to initiating treatment with this drug, the treating physician has at least excluded each of the following: (i) malnutrition, (ii) hypopituitarism, (iii) hypothyroidism, (iv) medication side effects; AND

The treatment must not be in a patient with known epiphyseal closure/growth plate fusion (i.e. the patient is known to have ceased growing); AND

Must be treated by a paediatric endocrinologist; the authority application must be completed by this physician type; or

Must be treated by a paediatrician who has consulted the above mentioned specialist type; the authority application must be completed by this paediatrician; AND

Patient must be aged from 2 years up until their 18th birthday.

An older child is defined as a male with a chronological age of at least 12 years or a bone age of at least 10 years, or a female with a chronological age of at least 10 years or a bone age of at least 8 years.

The initial treatment authority application must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail and must include the following:

(1) Insulin-like growth factor-1 deficiency:

State each of: (a) the patient's most recent basal IGF-1 level measured (ng/mL), (b) the measurement date (dd/mm/yy), (c) the name of the pathology result provider;

(2) Short stature:

State the patient's height (cm);

(3) Normal growth hormone levels:

State the patient's most recent growth hormone level measurement (mcg/L) - this figure must be greater than 6.6 mcg/L;

(4) Bone age: (where the patient has a chronological age of at least 2.5 years):

State each of: (a) the patient's bone age in numerical figures at the time when it was most recently determined, (b) the date (dd/mm/yy) of this determination that is within 12 months of this authority application;

(5) The patient's weight (kg);

(6) The prescribed dose (mg/kg) (between 0.04 to 0.12);

(7) The number of vials rounded to the nearest whole number, to provide sufficient drug quantity for 30 days of treatment per dispensing - see the relevant 'NOTE' attached to this listing for guidance.

Height, growth velocity and weight measurements must not be more than three months old at the time of application.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Severe growth failure with primary insulin-like growth factor-1 deficiency

Continuing treatment

Patient must have previously received PBS-subsidised treatment with this drug for this condition; AND

Patient must have a bone age of less than 13.5 years (females); or

Patient must have a bone age of less than 15.5 years (males); AND

The treatment must not be in a patient with known epiphyseal closure/growth plate fusion (i.e. the patient is known to have ceased growing); AND

The condition must be responsive to this drug treatment as evidenced by each of: (i) patient is showing catch-up for height standard deviation score against Laron syndrome (growth hormone insensitivity syndrome) growth charts, (ii) patient has a growth velocity of greater than 2 cm per year (extrapolated for time on treatment) at the time of this continuing authority application; or

The condition must be yet to respond to this drug treatment only for the reason of sub-optimal dosing; AND

Must be treated by a paediatric endocrinologist; the authority application must be completed by this physician type; or

Must be treated by a paediatrician who has consulted the above mentioned specialist type; the authority application must be completed by this paediatrician; AND

Patient must be aged from 2 years up until their 18th birthday.

The continuing treatment authority application must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail and must include:

(1) The patient's height (cm);

(2) Where this authority application seeks to continue treatment where there has been an inadequate response to treatment due to sub-optimal dosing, state each of:

(i) the most recently prescribed dose (mg/kg) that resulted in an inadequate response;

(ii) the dose (mg/kg) (between 0.04 to 0.12) that was/will be subsequently prescribed to address the inadequate response;

(3) The patient's weight (kg);

(4) The patient's growth velocity in response to the preceding supply of drug (cm/year; extrapolated for time on treatment);

(5) The number of vials rounded to the nearest whole number, to provide sufficient drug quantity for 30 days of treatment per dispensing - see the relevant 'NOTE' attached to this listing for guidance.

Height, growth velocity and weight measurements must not be more than three months old at the time of application.

Document growth improvements in the patient's medical records.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).